Game Changer: Larry Steinman, MD

We need to kill cells that attack the pancreas but leave the rest of the immune cells to keep us healthy.

Larry Steinman, MD, researching how to prevent type 1

The research Larry Steinman, MD, has devoted himself to for 15 years might change your life. His area of study? A “vaccine” with the potential to turn off the immune cells that attack the pancreas and cause type 1 diabetes.

Past research has looked at drugs that can suppress the immune system enough to cure diabetes. But there’s a catch: In destroying the immune cells that attack the pancreas, the treatments also take out helpful cells that prevent illness. “We need to kill cells that attack the pancreas but leave the rest of the immune cells to keep us healthy,” says Steinman, 65, professor of pediatrics and neurology at Stanford University. His approach specifically targets the rogue cells attacking the pancreas, leaving the rest of the body’s immune cells alone.

His most recent study, a Phase 2a clinical trial, looked at the safety of an experimental medication in adults who had been diagnosed with type 1 diabetes within the past five years. They were injected with the drug for 12 weeks as Steinman and his colleagues monitored their C-peptide levels. The higher a person’s C-peptide level, the better his or her beta cell function or the more beta cells are still alive and potentially producing insulin. “To our surprise and delight, we not only saw the procedure was safe, but during the time we were giving the [injections] there was an increase in C-peptide,” Steinman says.

The groundbreaking results are the first from the biopharmaceutical company Tolerion, which Steinman cofounded with fellow researchers and launched this past spring. Next up: a larger trial with kids and adults to see the effects of the injections on C-peptide levels, A1C, and insulin use. If all goes well with this new, Phase 2b trial, Steinman and his fellow researchers will launch a Phase 3 study on the drug before submitting it for Food and Drug Administration approval. “If the results [of the Phase 2b trial] are extraordinary, we could petition for an early approval [by the FDA],” he says.

How the medication would be used is yet to be determined. “It won’t work like we usually think about a vaccine,” Steinman says. That is, children wouldn’t be vaccinated for diabetes to prevent the condition from ever developing. At this stage in his research, Steinman says the drug would seem to work best as periodic injections, which people with diabetes would take less frequently than their current insulin doses. (Steinman says it’s too early to know how long each shot would last, but he envisions people injecting once every couple of weeks, once a month, or, possibly in the future, once a year.)

“I can’t imagine there’s any person in the world who wouldn’t say, ‘Are you kidding? Of course I’d get a shot every two weeks,’ ” he says. “Down the road, you’d have a much better shot of not having retinal damage, nerve damage, [or other complications]. Those benefits are huge.”